Gene therapy is one of the most promising technologies of modern medicine. Gene therapy is the treatment that consists of introducing into a patient a normal copy of one or more defective genes responsible for the patient’s disease. The most commonly used vector in gene therapy is the adenoviral vector. Clinical use of this vector has been limited, since conventional production systems produce material that is contaminated with unwanted replication competent adenoviruses (RCA). Due to its genetic design, Crucell’s PER.C6^® technology has eliminated this problem. PER.C6^® cells do not generate RCA, making it the state-of-the-art platform for the large -scale production of adenoviral vectors for clinical applications.

PER.C6^® cells support high yield RCA-free vector production Crucell has developed a plasmid system that allows easy insertion of heterologous genes into the adenovirus serotype-5 backbone. The shuttle vector used for the insertion of the therapeutic genes is marketed under the trademark pAdApt™. The pAdApt™ vector is specifically tailored to work with PER.C6^® technology and enables the generation of recombinant replication–deficient adenovirus vectors that are free of RCA that results from homologous recombination.

Crucell owns fully operational BSL-II laboratories with integrated upstream and downstream capacities. These units can be used to produce and purify recombinant adenovirus research batches. Crucell offers a routine in-house program to generate and produce small-scale vector batches for research purposes. The delivery of a purified vector research batch takes 4 months from start to finish allowing for rapid proof of concept studies. Over the last 5 years, Crucell has produced hundreds of research batches that on average contained more than 1 x 10¹¹ virus particles per milliliter of highly purified replication deficient vector particles. In addition, Crucell offers the manufacturing of gene therapy products for clinical testing. These batches are manufactured in its GMP production facilities on the PER.C6^® cell line in bioreactors at 20L scale all with consistently high quality and yields.  At present, Crucell is expanding its GMP-production capacity to >100L working volumes.